Table 1 Patient and HCT characteristics.

Total n = 89	n (%)		n (%)
Center		Conditioning regimen
Leiden University Medical Center	12 (13.5)	Cy/Flu	51 (57.3)
Memorial Sloan Kettering Cancer Center	35 (39.3)	Bu^d/Cy/Flu	26 (29.2)
University Medical Center Utrecht/Princess Maxima Center for Pediatric Oncology	42 (47.2)	TBI/Cy/Flu	11 (12.4)
Age at HCT		Cy/Thiotepa	1 (1.1)
Median (years)	9.2	Serotherapy
Range (years)	1.7-44	ATG	84 (94.4)
Gender		Alemtuzumab	1 (1.1)
Female	33 (37.1)	None	4 (4.5)
Male	56 (62.9)	HLA matching
Complementation group		Matched	59 (66.3)
FANCA	52 (58.4)	Mismatched	30 (33.7)
FANCC	22 (24.7)	Donor
FANCE	2 (2.2)	MUD	37 (41.6)
FANCG	3 (3.4)	MRD	22 (24.7)
Other^a	4 (4.5)	MMUD	20 (22.5)
Unknown	6 (6.7)	MMRD	10 (11.2)
Disease status at time of HSCT		Stem cell source
Bone marrow failure +/− cytogenetic changes^b	70 (78.7)	Bone marrow	45 (50.6)
MDS/AML^c	19 (21.3)	Peripheral blood	32 (36)
Follow-up		Cord blood	12 (13.5)
Median (years)	3.6	Graft manipulation
Range (years)	0.9–14.3	Unmanipulated/conventional^e	52 (58.4)
		Ex-vivo T cell depletion^f	37 (41.6)

^aIn the group ‘Other’ we included one of each of the following: FANCB, FANCD2, FANCL, and FANCM.
^bCytogenetic changes observed in patients with bone marrow failure were: 3q26/EVI1 rearrangement, +X, 7-, 7q-, and 20q-.
^cCytogenetic changes observed in patients with MDS/AML were: 3q26/EVI1 rearrangement, 1q+, 1q24+, 1q25+, 3q27+, 6p25-, 7q-, 9-, 9+, 11q-, 12p-, 12p13-, 15q+.
^dBU target was 18–22 mg*h/L at MSKCC and 30 mg*h/L at LUMC and UMCU/PMC.
^eGvHD prophylaxis used was dependent on graft source/manipulation. For bone marrow we used cyclosporine (CsA) and methotrexate (n = 31), CsA and mycophenolate mofetil (MMF; n = 3), CsA/MMF/prednisone (n = 1), CsA/MMF/tacrolimus/prednisone (n = 1), CsA/methotrexate/tacolimus (n = 1), MMF/prednisone (n = 1), MMF alone (n = 1), or CsA alone (n = 1). For cord blood we used CsA/prednisone (n = 9) or CsA alone (n = 3).
^fT-cell depletion devices used were Isolex (n = 8) and CliniMACS (n = 29).

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